16 July 2024 Leiden, The Netherlands, 16 July 2024 – Azafaros B.V. today announced positive topline data from its RAINBOW study, a Phase 2 clinical trial investigating nizubaglustat in patients with a genetic diagnosis of either GM2 gangliosidosis or Niemann-Pick disease type C (NPC). About the Trial The trial was conducted across three sites in Brazil, …
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Niemann-Pick Type C News
Azafaros announces completion of Phase 2 RAINBOW study evaluating nizubaglustat in NPC and other rare disease patients
Leiden, The Netherlands, March 12, 2024 – Azafaros B.V. today announced the completion of its 12-week Phase 2 clinical study, RAINBOW (Phase 2 RAINBOW study NCT05758922). The randomized, double-blind, placebo-controlled study, conducted in Brazil, involved 13 patients older than 12 years who are affected by GM2 gangliosidosis or Niemann-Pick disease type C (NPC). The aim of the RAINBOW study is …
IntraBio files New Drug Application for N-acetyl-L-leucine (IB1001) for the treatment of NPC
IntraBio Inc. is pleased to share that the New Drug Application (NDA) for N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease type C (NPC) was submitted to the US Food and DrugAdministration (FDA) in January 2024. The FDA has a 60-day filing review period to determine whetherthe NDA is complete and accepted for review. With …